CAR-T(CAR-T)细胞疗法在非肿瘤性疾病中的应用
Chimeric antigen receptor T (CAR-T) cell therapy in non-oncological diseases.
CAR-T(CAR-T)细胞在血液系统恶性肿瘤中的应用推动了这种免疫治疗形式的显著进展。
CELL INTELLIGENCE · 肿瘤细胞治疗研究
肿瘤细胞治疗研究
英文原题:Cost-effectiveness of polatuzumab vedotin combined with chemoimmunotherapy in untreated diffuse large B-cell lymphoma.
Cost-effectiveness of polatuzumab vedotin combined with chemoimmunotherapy in untreated diffuse large B-cell lymphoma.
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在初治弥漫性大B细胞淋巴瘤(DLBCL)患者中,POLARIX研究(一项比较维泊妥珠单抗联合利妥昔单抗-环磷酰胺、多柔比星和泼尼松[R-CHP]与利妥昔单抗-环磷酰胺、多柔比星、长春新碱和泼尼松[R-CHOP]在弥漫性大B细胞淋巴瘤受试者中的疗效和安全性的研究)报告,与标准利妥昔单抗、环磷酰胺、多柔比星、长春新碱和泼尼松(R-CHOP)相比,使用维泊妥珠单抗、利妥昔单抗、环磷酰胺、多柔比星和泼尼松(pola-R-CHP)的2年无进展生存期(PFS)改善了6.5%,总生存期(OS)或安全性无差异。
我们评估了pola-R-CHP用于DLBCL的成本效益。我们通过开发Markov模型(终身时间范围),使用一系列合理的长期结局,对假设的美国成人队列(平均年龄65岁)初治DLBCL患者进行建模,以模拟pola-R-CHP和R-CHOP的成本效益。进展率和OS根据POLARIX估算。结局指标以增量成本效益比报告,支付意愿(WTP)阈值为每质量调整生命年(QALY)150 000美元。假设pola-R-CHP的5年PFS为69.6%,R-CHOP为62.7%,pola-R-CHP在WTP为150 000美元时具有成本效益(增量成本效益比,84 308美元/QALY)。如果pola-R-CHP的5年PFS为66.1%或更低,则不再具有成本效益。单因素敏感性分析显示,在WTP为150 000美元时,pola-R-CHP在成本高达276 312美元时仍具有成本效益。在WTP为150 000美元时,pola-R-CHP在10 000次Monte Carlo迭代中有56.6%为成本效益策略。如果PFS的绝对获益随时间维持,在$150 000/QALY的WTP下,pola-R-CHP相比R-CHOP具有成本效果。
然而,其成本效果高度依赖于其长期结局和CAR-T 细胞治疗的成本。常规使用pola-R-CHP将显著增加医疗支出。降价或识别获益最大的亚组将改善成本效果。
In patients with treatment-naive diffuse large B-cell lymphoma (DLBCL), the POLARIX study (A Study Comparing the Efficacy and Safety of Polatuzumab Vedotin With Rituximab-Cyclophosphamide, Doxorubicin, and Prednisone [R-CHP] Versus Rituximab-Cyclophosphamide, Doxorubicin, Vincristine, and Prednisone [R-CHOP] in Participants With Diffuse Large B-Cell Lymphoma) reported a 6.
5% improvement in the 2-year progression-free survival (PFS), with no difference in overall survival (OS) or safety using polatuzumab vedotin, rituximab, cyclophosphamide, doxorubicin, and prednisone (pola-R-CHP) compared with standard rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP).
We evaluated the cost-effectiveness of pola-R-CHP for DLBCL.
We modeled a hypothetical cohort of US adults (mean age, 65 years) with treatment-naive DLBCL by developing a Markov model (lifetime horizon) to model the cost-effectiveness of pola-R-CHP and R-CHOP using a range of plausible long-term outcomes. Progression rates and OS were estimated from POLARIX. Outcome measures were reported in incremental cost-effectiveness ratios, with a willingness-to-pay (WTP) threshold of $150 000 per quality-adjusted life-year (QALY). Assuming a 5-year PFS of 69. 6% with pola-R-CHP and 62.
7% with R-CHOP, pola-R-CHP was cost-effective at a WTP of $150 000 (incremental cost-effectiveness ratio, $84 308/QALY). pola-R-CHP was no longer cost-effective if its 5-year PFS was 66. 1% or lower. One-way sensitivity analysis revealed that pola-R-CHP is cost-effective up to a cost of $276 312 at a WTP of $150 000.
pola-R-CHP was the cost-effective strategy in 56. 6% of the 10 000 Monte Carlo iterations at a WTP of $150 000. If the absolute benefit in PFS is maintained over time, pola-R-CHP is cost-effective compared with R-CHOP at a WTP of $150 000/QALY.
However, its cost-effectiveness is highly dependent on its long-term outcomes and costs of chimeric antigen receptor T-cell therapy. Routine usage of pola-R-CHP would add significantly to health care expenditures. Price reductions or identification of subgroups that have maximal benefit would improve cost-effectiveness.
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